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Design of synthetic materials for intracellular delivery of RNAs: From siRNA-mediated gene silencing to CRISPR/Cas gene editing

  • Jason B. Miller
  • Daniel J. Siegwart
Review Article
  • 61 Downloads

Abstract

Ribonucleic acids (RNAs) possess great therapeutic potential and can be used to treat a variety of diseases. The unique biophysical properties of RNAs, such as high molecular weight, negative charge, hydrophilicity, low stability, and potential immunogenicity, require chemical modification and development of carriers to enable intracellular delivery of RNAs for clinical use. A variety of nanomaterials have been developed for the effective in vivo delivery of short/small RNAs, messenger RNAs, and RNAs required for gene editing technologies including clustered regularly interspaced palindromic repeat (CRISPR)/Cas. This review outlines the challenges of delivering RNA therapeutics, explores the chemical synthesis of RNA modifications and carriers, and describes the efforts to design nanomaterials that can be used for a variety of clinical indications.

Keywords

nucleic acid therapeutics nanoparticles synthetic nanomaterials RNAi mRNA CRISPR/Cas 

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Notes

Acknowledgements

D. J. S. acknowledges financial support from the Welch Foundation (I-1855), American Cancer Society (RSG-17-012-01), Department of Defense (CA150245P3), and Cancer Prevention and Research Institute of Texas (CPRIT) (R1212).

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Copyright information

© Tsinghua University Press and Springer-Verlag GmbH Germany, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Simmons Comprehensive Cancer CenterUniversity of Texas Southwestern Medical CenterDallasUSA
  2. 2.Department of BiochemistryUniversity of Texas Southwestern Medical CenterDallasUSA

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